Corporate Presentation
September 2014
Forward-looking Statements
Certain statements contained herein including, but not limited to, statements related to
anticipated timing of initiation and completion of clinical trials, anticipated size of clinical trials,
therapeutic and market potential of XOMA’s product candidates, continued sales of approved
products, regulatory approval of unapproved product candidates, sufficiency of our cash
resources and anticipated levels of cash utilization, or that otherwise relate to future periods
are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933
and Section 21E of the Securities Exchange Act of 1934.
These statements are based on assumptions that may not prove accurate, and actual results
could differ materially from those anticipated due to certain risks inherent in the biotechnology
industry and for companies engaged in the development of new products in a regulated
market. Potential risks to XOMA meeting these expectations are described in more detail in
XOMA's most recent filing on Form 10-K and in other SEC filings. Consider such risks
carefully when considering XOMA's prospects. Any forward-looking statement herein
represents XOMA’s views only as of the date of this presentation and should not be relied upon
as representing its views as of any subsequent date. XOMA disclaims any obligation to update
any forward-looking statement, except as required by applicable law.
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XOMA: Value Creation and Value Capture
 Create value by focusing on lead asset ─ gevokizumab
• Potent, fully humanized allosteric-modulating monoclonal antibody
• Once-a-month, subcutaneous injection
• Modulates inflammatory response to cytokine interleukin-1 beta (IL-1 β)
Inflammatory diseases
induced through
over-produced IL-1β by
multiple Immune
system cells
IL-1β binds to
IL-1β Receptor
Gevokizumab
binds to IL-1β
and
down-modulates
IL-1β signaling
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Disruption of
disease-causing
inflammatory cascade
XOMA: Value Creation and Value Capture
 Create value by developing gevokizumab broadly across multiple
inflammatory indications
• Execute ongoing Phase 3 EYEGUARD™ studies in non-infectious uveitis (NIU)
– Report top-line results from EYEGUARD-B, Behçet's uveitis patients, approximately 6
weeks after achieving the target event
– EYEGUARD-A and EYEGUARD-C studies in broad NIU population - results will follow
• Launch Phase 3 studies in next pivotal indication: pyoderma gangrenosum
• Use expansive proof-of-concept (POC) program to let gevokizumab lead us to
next diseases for pivotal development
– Multiple indications in POC testing at XOMA
– Leveraging Servier’s development in additional POC indications
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XOMA: Value Creation and Value Capture
 Capture value for XOMA by commercializing gevokizumab
directly in the U.S.
• Deal with Servier retains XOMA U.S. and Japanese commercial rights for
gevokizumab
• Select indications served by specialty physicians to enable direct
commercialization by XOMA in the U.S.
• Leverage Servier commercialization outside the U.S. and Japan; milestones
and royalties to XOMA
• License gevokizumab to a Japanese development and commercialization
partner
 Use gevokizumab success to advance additional XOMA
discoveries
• 30 years of leadership in antibody discovery and development
• Sizeable portfolio of potential antibodies to explore
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Gevokizumab: Pipeline in a Product
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Upcoming
Milestones
XOMA’s Development Programs
Non-infectious uveitis (NIU) – EYEGUARD™-A & -C*
Pyoderma gangrenosum (PG)
Multiple POC Programs
2014/2015:
Ph 3 Endpoints
Comments on final
protocols received
As Occurring
Servier’s Development Programs
Behçet's uveitis - EYEGUARD-B
Cardiovascular
Multiple POC programs
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*Worldwide development by both XOMA and Servier
Q3 14:
Ph 3 Endpoint
Ongoing
As Occurring
Gevokizumab: Non-infectious Uveitis (NIU)
Gevokizumab: Three EYEGUARD™ Phase 3
Studies Ongoing in Non-infectious Uveitis
 Inflammation of the uvea which can
occur in front, middle and/or back of
the eye
• Can be infectious or non-infectious
• Gevokizumab targeted for non-infectious
patients where back of the eye is affected
 150,000 NIU patients in U.S. is
significant market opportunity yet
retains orphan status (granted)
 Multiple etiologies
• Behçet's uveitis is representative of multiple
forms
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Gevokizumab: Clinical Activity in XOMA’s First
Phase 2 Trial of Behçet's Subset of NIU
 7 of 7 patients showed rapid treatment effect for intraocular inflammatory attack
 5 of 5 responded to second dose
hypopyon
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off-center iris
* photon counts per millisecond
Day 0:
Day 1:
Day 4:
Flare score: 467 ph/msec *
Anterior chamber cell: 4+
Vitreous haze: 3+
Fundus score: 8
Flare score: 45 ph/msec
Anterior chamber cell: 4+
Vitreous haze: 3+
Fundus score: 4
Flare score: 62 ph/msec
Anterior chamber cell: 4+
Vitreous haze: 2+
Fundus score: 4
Gevokizumab: Phase 2 Behçet's Uveitis Studies
Clearance of Vitreous Haze
 Patient from XOMA 7-patient Phase 2 study
Day 0 - Vit. Haze: 3+
Day 1 - Vit. Haze: 3+
Day 4 - Vit. Haze: 2+
Day 7 - Vit. Haze: 1+
Day 14 - Vit. Haze: 0.5+
Day 21 - Vit. Haze: 0
 Servier’s study in 21 patients with Behçet's uveitis patients included 15
patients with acute disease
• All responded to gevokizumab - most within 1 week
• Of 11 acute patients with a baseline vitreous haze score of ≥ 2+, 8 showed at least a
2-unit reduction at Day 70, remaining 3 had at least a 1-unit reduction
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Gevokizumab: Phase 3 EYEGUARD™ Program
Study Designs
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EYEGUARD-A
(XOMA)
EYEGUARD-B
(SERVIER)
EYEGUARD-C
(XOMA)
Active/Controlled
Active
Controlled
Controlled
# Patients
300
Up to 110
300
# Sites
140
44
140
Gevokizumab doses
(Monthly)
30mg, 60mg
Placebo
60mg
Placebo
30mg, 60mg
Placebo
Randomization
1:1:1
1:1
1:1:1
Start date
June 2012
September 2012
October 2012
Estimated primary
endpoint achievement
Year-end 2014
Q3 2014
Early 2015
Gevokizumab: EYEGUARD-B Study Design and
Status
 Ex-U.S. Behçet's uveitis patients, randomized, double-masked
 Receive 60mg gevokizumab or placebo subcutaneously monthly
• Randomized 1:1
 Patients must be stable on corticosteroids prior to
randomization
 Steroids further tapered according to a pre-set fixed schedule
 Primary endpoint is time to first exacerbation which is
interpolated from Kaplan-Meier survival analysis
 Hazard ratio of 0.3 gives ninety percent power at p≤ 0.05
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Gevokizumab: Driving Toward First BLA in
Behçet’s Uveitis
 EYEGUARD-B results are expected first
• Servier reached targeted enrollment in Q2
• Waiting for final pre-specified exacerbations
 Use EYEGUARD-B results to request pre-BLA Meeting with FDA
 Perform statistical analysis of XOMA and Servier Phase 2 results
to support EYEGUARD-B results
 Initiate EYEGUARD-US study in up to 28 Behçet's uveitis patients
to further supplement overall results
• May not be necessary for approval
• Interim results may be used for informational purposes
• Pivotal results may be required for approval
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Gevokizumab: EYEGUARD-US Behçet’s Uveitis
Supplemental Study
 Enrolling patients with either active or controlled Behçet’s
Uveitis
• Patients with active disease are brought under control with gevokizumab
(open label)
 Patients can be under control with other therapies
• Treatment control transferred to gevokizumab
– From Infliximab (anti-TNF-a)
– From Prednisone and/or other immunosuppressives
 Once patients are controlled on gevokizumab for 28 days • Half of the patients continue to receive monthly 60mg subcutaneous
treatment with gevokizumab
• Half of the patients receive placebo
 Primary Endpoint is time to first ocular exacerbation
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Gevokizumab: Pyoderma Gangrenosum
(PG)
Pyoderma Gangrenosum: XOMA’s Next
Phase 3 Indication
 Severe inflammatory, ulcerative disease of the skin with
undetermined cause
• One of several indications collectively known as neutrophilic dermatoses
• Claims data over past 3 years indicate U.S. pyoderma gangrenosum patient
population of 11,000 - 14,000
• Typically takes >11 months to fully heal with corticosteroids*
• Orphan Drug Designation granted by FDA: February 2014
 6 patients enrolled in pilot study at 60mg dose
• 5 patients showed improvement in ulcer size by Day 28
– 4 patients had total resolution of ulcer by Day 84
• All patients demonstrated pain improvement
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*Bennett ML et al. “Pyoderma gangrenosum. A comparison of typical and atypical forms with an emphasis on time to remission. Case review of 86 patients
from 2 institutions.” Medicine (Baltimore) Jan;79(1):37-46. PMID: 10670408
Gevokizumab: Pyoderma Gangrenosum
Subject 2300
DAY 0
DAY 84
1
1
Ulcer Size:
ID
1
17
Day 0
(cm2)
Area
5.2
Pain
(0 - 10)
Day 84
Area
(cm2)
0
D%
100
Day 0
Day 84
Investigators Assessment of Target Ulcer
10
7
0: Total resolution of target ulcer with no sign
of active PG
Gevokizumab: Pyoderma Gangrenosum
Subject 2802
DAY 0
DAY 84
1
1
Ulcer Size:
ID
1
18
Day 0
Day 84
Area (cm2)
16
Area (cm2)
0
Pain
(0 - 10)
D%
100
Day 0
4
Day 84
0
Investigators Assessment of Target Ulcer
0: Total resolution of target ulcer with no sign
of active PG
Gevokizumab: Percent Clearance of Target
Ulcer in Pyoderma Gangrenosum Patients
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Gevokizumab: Pyoderma Gangrenosum Phase 3
Program Study Designs
Pyoderma Gangrenosum
Phase 3 Studies
U.S. Only
(Study 172)
U.S. & x-U.S.
(Study 173)
# Patients
58
58
Gevokizumab doses
(Monthly)
60mg : Placebo
60mg : Placebo
Randomization
1:1
1:1
Primary endpoint
Complete healing of target ulcer at approximately Day 124
Secondary endpoints
Multiple, including time to ulcer closure and pain
Statistical Powering
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80%; p=0.05
80%; p=0.05
XOMA Metabolic (XMet) Program
Creating Value from XMet Discovery Program
 XMet A: selective insulin receptor modulator (SIRM)
• May provide a long-acting alternative to basal insulin*
• Data from non-human primates should be predictive and is being used for
partnering discussions
 XMet S: insulin sensitizing antibodies
• May enable more effective use of own insulin for early stage diabetics
• Mouse model has shown a 20x increase in sensitivity to insulin
 XMet D: antagonist (deactivator) of the insulin receptor
• 3 potential rare disease indications to be evaluated:
– Congenital Hyperinsulinism (CHI)
– Insulinomas
– Hyperinsulinemic Hypoglycemia Post Gastric Bypass Surgery
• Patients concentrated in a few centers nationwide - development could be
rapid
• IND to be filed in 2014
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*Vigneri, R., Squatrito, S., Frittitta, L., Diabetes May 2012
Building Value Through Development Activities
Compound
Indication
XOMA’s Development Programs
Gevokizumab
Non-infectious uveitis (NIU) – EYEGUARD-A & -C
Pyoderma gangrenosum (PG)
Erosive osteoarthritis of the hand (EOA)
Moderate-to-severe acne
Non-infectious scleritis
Autoimmune inner ear disease (AIED)
XMet D
Excess insulin – Rare disease indications
Servier’s Development Programs
Gevokizumab
Behçet's uveitis - EYEGUARD-B
Cardiovascular
Polymyositis/dermatomyositis
Schnitzler syndrome
Giant cell arteritis (GCA)
XOMA Development Activities for Licensure
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XMet A
Type 1 & 2 diabetes mellitus
XMet S
Type 2 diabetes mellitus
Preclinical
Phase 1
Phase 2
Phase 3
Marketed
Financial Highlights
 $75.9 million cash at June 30, 2014
 Approximately 107 million shares outstanding
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Investment Thesis –
Committed to Become a U.S. Commercial Company
 Complete gevokizumab global Phase 3 program in non-infectious
uveitis (NIU) clinical trials
• EYEGUARD-B data may allow a “Behçet's first” BLA submission
• Initiate EYEGUARD-US study to supplement Behçet's uveitis BLA submission
• EYEGUARD-A and -C may expand label to broader NIU population
 Launch gevokizumab pyoderma gangrenosum pivotal program
 Assess gevokizumab’s potential in additional inflammatory
indications through POC program
 Advance proprietary allosteric modulating antibody pipeline
towards an IND and commercialization or licensure, particularly
XMet platform
 Use gevokizumab to build XOMA into a fully integrated company
with a commercial capability
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